Nov. 12--The Muscular Dystrophy Association is prepared to underwrite a clinical trial for a promising gene therapy that could preserve and strengthen muscle fibers in patients with neuromuscular diseases.
Based on reports published Wednesday in Science Translational Medicine, injections of genes for a protein called follistatin increased muscle size and strength in the upper legs of four macaque monkeys.
The tests were conducted by a team headed by neurologist Jerry Mendell and research scientist Brian Kaspar of Nationwide Children's Hospital in Columbus, Ohio.
The therapy, even if proved successful in human trials, would not be a cure for muscular dystrophy, said Dr. Valerie Cwik, medical director and executive vice president for research at MDA headquarters in Tucson.
Muscular dystrophy is the name given to a group of genetic disorders that cause muscle weakening and loss over time.
"Follistatin therapy wouldn't actually solve the underlying problem, but what it could do is help preserve and help strengthen the muscle fibers that are there and maintain strength and function longer," Cwik said.
"It would not correct the muscle degeneration that is occurring, but the muscle fibers that are there could get stronger."
Cwik said the MDA has invited Mendell to submit a request for funding of human clinical trials based on his results.
Those trials wouldn't occur any time soon, Cwik said.
"Before it could be used in people, there is a lot of testing that needs to be done," she said.
Cwik said Mendell "has been working toward finding a therapy for most of his career. He is very dedicated to this field."
Follistatin works by inhibiting the effect of another protein called myostatin, which restricts muscle growth, Cwik said.
The therapy "could be used in a wider variety of muscle diseases," Cwik said.
According to a release from MDA headquarters in Tucson: "The findings could have implications particularly for injured and aging people worldwide, and for tens of millions experiencing muscle loss associated with cancer, AIDS and muscle diseases."
"It would not correct the muscle degeneration that is occurring, but the muscle fibers that are there could get stronger."
Dr. Valerie Cwik
MDA medical director
Contact reporter Tom Beal at 573-4158 or tbeal@azstarnet.com
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